The family of a Spring Lake 9-year-old said their daughter has shown improvements following her participation in a clinical trial for sickle cell disease gene therapy.
Leilani Beasley has more energy since undergoing the treatment, which is one of the new therapies already approved by the FDA for patients 12 and older.
“It does include chemotherapy to eliminate the natural stem cells that come into the hospital to get that process. They would then get an infusion of their own stem cells back that have been modified and then their body will recover from that chemotherapy, from that infusion or the delivery of the new cells until they start to take over and do their job,” said Dr. Jennifer Rothman, the Director of the Pediatric Sickle Cell Program at Duke.
Sickle cell disease impacts about 100,000 Americans, about 90% of whom are Black. The disease is hereditary; Leilani’s father Carlton also has it.
“You have to be born with it, and you live the rest of your life with it,” Carlton said.